ALN-AS1: Phase I data

Data from the single-blind, placebo-controlled first part of a European Phase I trial in 16 asymptomatic “high excreter” AIP patients with elevated levels of ALA and PBG showed that single subcutaneous doses of 0.035, 0.1, 0.35 and 1 mg/kg ALN-AS1 were generally well tolerated with no treatment-related serious adverse events reported. Additionally, ALN-AS1 led to mean maximum reductions in ALA levels of 44.4% at the 0.035 mg/kg dose, 65.5% (p<0.05) at the 0.1 mg/kg dose and 77.1% (p<0.01) at the 0.35 mg/kg dose vs. 8.5% for placebo. ALN-AS1 led to mean maximum reductions in PBG levels of 45.3% at the 0.035 mg/kg dose, 70.5% (p<0.05) at the 0.1 mg/kg dose and 72.6% (p=0.06) at the 0.35 mg/kg dose vs. 11.7% for placebo. ...